CN103764824B - 粘多糖贮积症和其它溶酶体病症的抗TNF-α疗法 - Google Patents

粘多糖贮积症和其它溶酶体病症的抗TNF-α疗法 Download PDF

Info

Publication number
CN103764824B
CN103764824B CN201280037341.5A CN201280037341A CN103764824B CN 103764824 B CN103764824 B CN 103764824B CN 201280037341 A CN201280037341 A CN 201280037341A CN 103764824 B CN103764824 B CN 103764824B
Authority
CN
China
Prior art keywords
disease
mps
purposes according
lysosomal storage
subject
Prior art date
Legal status (The legal status is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the status listed.)
Active
Application number
CN201280037341.5A
Other languages
English (en)
Chinese (zh)
Other versions
CN103764824A (zh
Inventor
爱德华·H·舒克曼
凯洛格拉·M·西蒙娜罗
加里·E·斯特赖克
海伦·维拉萨拉
Current Assignee (The listed assignees may be inaccurate. Google has not performed a legal analysis and makes no representation or warranty as to the accuracy of the list.)
Icahn School of Medicine at Mount Sinai
Original Assignee
Mount Sinai School of Medicine
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Mount Sinai School of Medicine filed Critical Mount Sinai School of Medicine
Publication of CN103764824A publication Critical patent/CN103764824A/zh
Application granted granted Critical
Publication of CN103764824B publication Critical patent/CN103764824B/zh
Active legal-status Critical Current
Anticipated expiration legal-status Critical

Links

Classifications

    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A61K38/465—Hydrolases (3) acting on ester bonds (3.1), e.g. lipases, ribonucleases
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00—Medicinal preparations containing organic active ingredients
    • A61K31/70—Carbohydrates; Sugars; Derivatives thereof
    • A61K31/715—Polysaccharides, i.e. having more than five saccharide radicals attached to each other by glycosidic linkages; Derivatives thereof, e.g. ethers, esters
    • A61K31/737—Sulfated polysaccharides, e.g. chondroitin sulfate, dermatan sulfate
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/43—Enzymes; Proenzymes; Derivatives thereof
    • A61K38/46—Hydrolases (3)
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K45/00—Medicinal preparations containing active ingredients not provided for in groups A61K31/00 - A61K41/00
    • A61K45/06—Mixtures of active ingredients without chemical characterisation, e.g. antiphlogistics and cardiaca
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P19/00—Drugs for skeletal disorders
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P43/00—Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00

Landscapes

  • Health & Medical Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • General Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Veterinary Medicine (AREA)
  • Public Health (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Animal Behavior & Ethology (AREA)
  • Epidemiology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Immunology (AREA)
  • General Chemical & Material Sciences (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Diabetes (AREA)
  • Organic Chemistry (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Molecular Biology (AREA)
  • Dermatology (AREA)
  • Obesity (AREA)
  • Hematology (AREA)
  • Endocrinology (AREA)
  • Emergency Medicine (AREA)
  • Physical Education & Sports Medicine (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines Containing Antibodies Or Antigens For Use As Internal Diagnostic Agents (AREA)
CN201280037341.5A 2011-06-20 2012-06-20 粘多糖贮积症和其它溶酶体病症的抗TNF-α疗法 Active CN103764824B (zh)

Applications Claiming Priority (5)

Application Number Priority Date Filing Date Title
US201161498946P 2011-06-20 2011-06-20
US61/498,946 2011-06-20
US201161569452P 2011-12-12 2011-12-12
US61/569,452 2011-12-12
PCT/US2012/043369 WO2012177778A1 (en) 2011-06-20 2012-06-20 Anti-tnf- therapy for the mucopolysaccharidoses and other lysosomal disorders

Publications (2)

Publication Number Publication Date
CN103764824A CN103764824A (zh) 2014-04-30
CN103764824B true CN103764824B (zh) 2018-04-24

Family

ID=47422922

Family Applications (1)

Application Number Title Priority Date Filing Date
CN201280037341.5A Active CN103764824B (zh) 2011-06-20 2012-06-20 粘多糖贮积症和其它溶酶体病症的抗TNF-α疗法

Country Status (6)

Country Link
US (1) US9155784B2 (https=)
EP (1) EP2721151B1 (https=)
JP (1) JP6175431B2 (https=)
CN (1) CN103764824B (https=)
ES (1) ES2664844T3 (https=)
WO (1) WO2012177778A1 (https=)

Families Citing this family (19)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US6849257B2 (en) 2000-02-04 2005-02-01 Children's Hospital Research Foundation Lipid hydrolysis therapy for atherosclerosis and related diseases
US8889127B2 (en) 2004-07-01 2014-11-18 Icahn School Of Medicine At Mount Sinai Targeted protein replacement for the treatment of lysosomal storage disorders
SI3998078T1 (sl) * 2009-08-28 2026-02-27 Genzyme Corporation Encimsko nadomestno zdravljenje z naraščajočim odmerkom za zdravljenje pomanjkanja kisle sfingomielinaze
FI3205351T3 (fi) 2010-04-23 2023-07-06 Alexion Pharma Inc Lysosomaalisen kertymätaudin entsyymi
KR20150038636A (ko) 2010-09-09 2015-04-08 시나게바 바이오파르마, 코포레이션 환자에서 리소좀 산 리파제 결핍증의 치료를 위한 리소좀 산 리파제의 용도
WO2012112681A1 (en) 2011-02-15 2012-08-23 Shire Human Genetic Therapies, Inc. Methods for treating lysosomal acid lipase deficiency
LT2753346T (lt) 2011-09-07 2020-08-10 Mount Sinai School Of Medicine Ceramidazė ir ląstelių diferenciacija
ES2897825T3 (es) 2012-06-01 2022-03-02 Icahn School Med Mount Sinai Niveles de ceramida en el tratamiento y prevención de infecciones
PT2968479T (pt) 2013-03-14 2019-08-07 Icahn School Med Mount Sinai Composições terapêuticas de ceramidase ácida e métodos de fabrico e utilização
SG11201509030TA (en) 2013-05-07 2015-11-27 Bio Blast Pharma Ltd Treatment of protein aggregation myopathic and neurodegenerative diseases by parenteral administration of trehalose
NZ720499A (en) * 2013-12-23 2020-04-24 Bcn Peptides Sa Bicalutamide analogs or (s)-bicalutamide as exocytosis activating compounds for use in the treatment of a lysosomal storage disorder or glycogenosis
SI2970413T1 (sl) * 2014-04-01 2018-10-30 Swedish Orphan Biovitrum Ab (Publ) Modificirana sulfamidaza in proizvodja le-te
US20150348259A1 (en) * 2014-06-03 2015-12-03 Carestream Health, Inc. Quantitative method for 3-d bone mineral density visualization and monitoring
WO2017019612A1 (en) * 2015-07-27 2017-02-02 Mary Kay Inc. Topical skin formulations
WO2020039480A1 (ja) 2018-08-20 2020-02-27 株式会社レクメド 新規ポリ硫酸ペントサンナトリウム製剤
FI3863647T3 (fi) * 2018-10-10 2024-03-01 Paradigm Biopharmaceuticals Ltd Pahanlaatuisen luustokivun hoitaminen pentosaanipolysulfaatilla
IL291799A (en) * 2019-10-01 2022-06-01 Seelos Therapeutics Inc Trehalose formulations and uses thereof
WO2021179047A1 (en) * 2020-03-12 2021-09-16 Cullis Hill Sydney David Treatment for coronavirus infection and associated cytokine toxicity
IT202200007808A1 (it) 2022-04-20 2023-10-20 Luigi Michele Pavone Composizioni terapeutiche per malattie causate da accumulo di eparan solfato

Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080292618A1 (en) * 2007-05-24 2008-11-27 The Regents Of The University Of California Intranuclear protein transduction through a nucleoside salvage pathway
US20100160253A1 (en) * 2007-05-31 2010-06-24 Glycan Biosciences Sulphated xylans for treatment or prophylaxis of respiratory diseases
US20110091442A1 (en) * 2009-10-19 2011-04-21 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders

Family Cites Families (22)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US5180715A (en) 1980-11-17 1993-01-19 The Regents Of The University Of California Irrigation of internal bladder surfaces in mammals with sodium pentosanpolysulfate
US5672347A (en) 1984-07-05 1997-09-30 Genentech, Inc. Tumor necrosis factor antagonists and their use
US5668116A (en) 1987-03-19 1997-09-16 Anthropharm Pty. Limited Anti-inflammatory compounds and compositions
US6451983B2 (en) 1989-08-07 2002-09-17 Peptech Limited Tumor necrosis factor antibodies
JP3443119B2 (ja) 1989-08-07 2003-09-02 ペプテック リミテッド 腫瘍壊死因子結合リガンド
US5656272A (en) 1991-03-18 1997-08-12 New York University Medical Center Methods of treating TNF-α-mediated Crohn's disease using chimeric anti-TNF antibodies
ES2121907T3 (es) 1992-08-28 1998-12-16 Bayer Ag Uso de anticuerpos monoclonales anti-tnf para el tratamiento de meningitis bacterianas.
CA2146647C (en) 1992-10-08 2009-05-05 Marc Feldmann Treatment of autoimmune and inflammatory disorders
US5643892A (en) 1995-06-07 1997-07-01 Baker Norton Pharmaceuticals, Inc. Method of treating chronic progressive vascular diseases
EP1005376B1 (en) 1997-03-14 2010-11-03 The Children's Hospital of Philadelphia Compositions for use in gene therapy for treatment of hemophilia
CA2319212A1 (en) 1998-02-17 1999-08-19 Genzyme Corporation Methods for purified aav vector production
WO1999057296A1 (en) 1998-05-01 1999-11-11 Genzyme Corporation Partially deleted adenoviral vectors
US7927587B2 (en) 1999-08-05 2011-04-19 Regents Of The University Of Minnesota MAPC administration for the treatment of lysosomal storage disorders
US20010034328A1 (en) 2000-02-25 2001-10-25 Cartt Stephen Lahue Treatment of male chronic pelvic pain syndrome
AU2001269923A1 (en) 2000-06-19 2002-01-02 Genzyme Corporation Combination enzyme replacement, gene therapy and small molecule therapy for lysosomal storage diseases
US7951545B2 (en) 2002-04-30 2011-05-31 Seikagaku Corporation Method for detecting lysosomal storage diseases
US7446098B2 (en) 2003-02-18 2008-11-04 Mount Sinai School Of Medicine Of New York University Combination therapy for treating protein deficiencies
US20050208090A1 (en) 2004-03-18 2005-09-22 Medtronic, Inc. Methods and systems for treatment of neurological diseases of the central nervous system
WO2007095688A1 (en) * 2006-02-23 2007-08-30 The Australian National University Methods for increasing the number of circulating cells
US20080076723A1 (en) 2006-09-27 2008-03-27 Sylvan Pharmaceuticals Pty Ltd. Inhibition of cathepsin K activity and the treatment and prevention of disease
US8540985B2 (en) * 2008-06-26 2013-09-24 Orphazyme Aps Use of Hsp70 as a regulator of enzymatic activity
CA3140959A1 (en) * 2009-11-27 2011-06-03 Genzyme Corporation An amorphous and a crystalline form of genz 112638 hemitartrate as inhibitor of glucosylceramide synthase

Patent Citations (3)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
US20080292618A1 (en) * 2007-05-24 2008-11-27 The Regents Of The University Of California Intranuclear protein transduction through a nucleoside salvage pathway
US20100160253A1 (en) * 2007-05-31 2010-06-24 Glycan Biosciences Sulphated xylans for treatment or prophylaxis of respiratory diseases
US20110091442A1 (en) * 2009-10-19 2011-04-21 Amicus Therapeutics, Inc. Novel compositions for preventing and/or treating lysosomal storage disorders

Non-Patent Citations (3)

* Cited by examiner, † Cited by third party
Title
Advantages of using same species enzyme for replacement therapy in a feline model of mucopolysaccharidosis type VI;Julie Bielicki et al;《THE JOURNAL OF BIOLOGICAL CHEMISTRY》;19991231;第274卷(第51期);摘要、36335页右栏第4段、36336页左栏第1段、36340页右栏第1段、36342页左栏第2段 *
Involvement of the Toll-like receptor 4 pathway and use of TNF-alpha antagonists for treatment of the mucopolysaccharidoses;Calogera M. Simonaro et al;《PNAS》;20100105;第107卷(第1期);摘要、第225页左栏第1段至第226页左栏第1段 *
Ultra orphan drugs for lysosomal storage disorders;bazian;《 Ultra orphan drugs for lysosomal storage disorders Online Report》;20091231;第3页 *

Also Published As

Publication number Publication date
JP6175431B2 (ja) 2017-08-02
CN103764824A (zh) 2014-04-30
WO2012177778A1 (en) 2012-12-27
US20140205584A1 (en) 2014-07-24
US9155784B2 (en) 2015-10-13
EP2721151A4 (en) 2015-04-22
EP2721151B1 (en) 2017-12-06
EP2721151A1 (en) 2014-04-23
ES2664844T3 (es) 2018-04-23
JP2014518222A (ja) 2014-07-28

Similar Documents

Publication Publication Date Title
CN103764824B (zh) 粘多糖贮积症和其它溶酶体病症的抗TNF-α疗法
US12036190B2 (en) Methods and compositions for the treatment of cytoplasmic glycogen storage disorders
Beck New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy
Ortolano et al. Treatment of lysosomal storage diseases: recent patents and future strategies
JP6251307B2 (ja) 酸性スフィンゴミエリナーゼ欠乏症に対する用量漸増酵素補充療法
Auclair et al. Replacement therapy in Mucopolysaccharidosis type VI: advantages of early onset of therapy
US20220251526A1 (en) Targeted therapeutic lysosomal enzyme fusion proteins, associated formulations and uses thereof
EP2977057A1 (en) Isolated recombinant human N-glycosylated lysosomal acid lipase.
Van Den Broek et al. Hurdles in treating Hurler disease: Potential routes to achieve a “real” cure
Byrne et al. Cipaglucosidase alfa plus miglustat: linking mechanism of action to clinical outcomes in late-onset Pompe disease
US20180326021A1 (en) Methods and Compositions for the Treatment of Cytoplasmic Glycogen Storage Disorders
WO2017049161A1 (en) ACID ALPHA-GLUCOSIDASE AND A β-2 AGONIST FOR THE TREATMENT OF LYSOSOMAL STORAGE DISORDER
WO2014120900A1 (en) Enhanced therapeutic regimens for treating fabry disease
EP3518962B1 (en) Sebelipase alfa for use in liver fibrosis and treating lysosomal acid lipase deficiency in patients based on ishak fibrosis stage
HK1197423B (en) Anti-tnf- therapy for the mucopolysaccharidoses and other lysosomal disorders
HK1197423A (en) Anti-tnf- therapy for the mucopolysaccharidoses and other lysosomal disorders
Beck Enzyme Replacement Therapy in Mucopolysaccharidosis Type II (Hunter Syndrome).
CN120731226A (zh) 用于预防和治疗患有黏多糖贮积症ii型的受试者的溶酶体酶缺乏的药物组合物和方法
Dierenfeld Enzyme replacement therapy treatment from birth increases therapeutic efficacy and generates tolerance to enzyme in canine mucopolysaccharidosis type I
Richard III Lysosomal storage disorders: current treatments and future directions
Pastores et al. Enzyme Replacement and Other Therapies for the Lysosomal Storage Disorders
Laron et al. Recommended by ISPED and SIMA
HK40001779A (en) Improved naglu fusion protein formulation
HK40001779B (en) Improved naglu fusion protein formulation
Richard Introduction to Biological and Small Molecule Drug Research and Development: Chapter 11. Lysosomal storage disorders: current treatments and future directions

Legal Events

Date Code Title Description
C06 Publication
PB01 Publication
C10 Entry into substantive examination
SE01 Entry into force of request for substantive examination
GR01 Patent grant
GR01 Patent grant